Cystic Fibrosis (CF) is a fatal disease caused by a defective gene that encodes for CF transmembrane conductance regulator (CFTR), a glycoprotein important in chloride transport. Approximately 75% of CF patients carry a deletion of phenylalanine from position 508 of the coding sequence for CFTR. This mutant CFTR is incompletely processed within the endoplasmic reticulum with limited transport of nonfunctinal CFTR to the airway epithelial surface, thereby causing defective chloride secretion. """"""""Protein assist"""""""" therapy, such as CPX is a new approach to activate chloride secretion via the mutant protein channels. CPX binds with high specificity and affinity to the first nucleotide binding domain of CFTR and restores function to mutant CFTR molecules in vitro. This Phase II study will evaluate the safety and efficacy of multiple ascending oral doses of oral CPX in adults with CF.

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
3M01RR000037-39S1
Application #
6263558
Study Section
Project Start
1998-12-01
Project End
1999-11-30
Budget Start
1998-10-01
Budget End
1999-09-30
Support Year
39
Fiscal Year
1999
Total Cost
Indirect Cost
Name
University of Washington
Department
Type
DUNS #
135646524
City
Seattle
State
WA
Country
United States
Zip Code
98195
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